Medical device stakeholders give House feedback on 4 separate bills

A variety of stakeholders in the medical device industry testified before the House Energy and Commerce Committee with respect to a number of bills related to devices, including the Over-the-Counter Hearing Aid Act (H.R.1652), the Medical Device Servicing Safety and Accountability Act (H.R. 2118), the Fostering Innovation in Medical Imaging Act of 2017 (H.R. 2009), and a bill to amend the Federal Food, Drug, and Cosmetic Act to improve the process for inspections of device establishments and for granting export certifications (H.R. 1736), which would enhance the authorization Medical Device User Fee Amendments (MDUFA IV). While stakeholders took opposing viewpoints with respect to some bills, those that spoke to MDUFA IV, including Director of the FDA’s Center for Devices and Radiological Health Jeffrey Shuren, all expressed their support.

Over-the-Counter Hearing Aid Act

The Over-the-Counter (OTC) Hearing Aid Act would permit the sale of OTC hearing aids for use in adults with mild to moderate hearing impairments. Speaking on behalf of the Hearing Industries Association (HIA), Dr. Thomas Powers of Powers Consulting, LLC supported the sales of OTC hearing aids for mild hearing impairments, but opposed the provision of such hearing aids for moderate use. Hearing impairment, particularly moderate and severe impairments, are complex and, in Powers’ opinion require consultation and fitting with hearing health professionals; in addition, he cited to a study indicating that hearing impaired individuals were more likely to be satisfied and wear hearing aids fitted by a professional.

Frank R. Lin, M.D., Ph.D., Associate Professor in the Departments of Otolaryngology-Head & Neck Surgery and Geriatric Medicine at the John Hopkins School of Medicine and in the Departments of Epidemiology and Mental Health at the Johns Hopkins Bloomberg School of Public Health, on the other hand, opined that OTC sales would make hearing aids more accessible and affordable to the hearing-impaired population; currently, less than 28 percent of the nearly 38 million Americans with significant hearing loss have access to the devices. He argued that alleged safety concerns about the sale of OTC hearing aids to individuals with moderate hearing loss were raised by parties looking to preserve the status quo, which offers little incentive for innovation of new market entry, with, “98 percent of the world’s hearing aid marketplace being controlled by six companies.” He likened the risks of OTC hearing aids to the risk of OTC reading glasses or aspirin.

Medical Device Servicing Safety and Accountability Act

H.R. 2118 would require establishments that service medical devices to register with the FDA, establish a complaint-handling system, and report adverse events to the agency. Joe Robinson, Senior Vice President of Health Systems Solutions for Philips North America, offered his support of the bill on behalf of the Medical Imaging & Technology Alliance (MITA). He noted that improper servicing of medical devices by non-manufacturer entities can injure patients via direct or indirect bodily harm, and can also cause problems for manufacturers down the road. For example, the manufacturer may not be aware of adverse events or may not be familiar with the chain of events that ultimately led to an adverse event, may experience difficulty upgrading parts that have been altered by a third party, or may have its device certification voided. MITA believes that registration and complaint handling is an important first step to improve service and safety.

Robert J. Kerwin, General Counsel for the International Association of Medical Equipment Remarketers and Services, Inc. (IAMERS), adamantly opposed the bill. He argued that the legislation provided a solution for a problem that does not exist, noting that, of the 177 public comments submitted to the FDA in response to its request for comments, including comments on service regulation, nearly none of them made negative comments about third-party device services, and that The Joint Commission, in its comment, stated that it “has no knowledge of any statistically significant level of safety problems resulting from the activities of any kind of maintenance/service provider.” Kerwin contrasted MITA’s opinion that increased regulation is necessary to prevent improper servicing with its members’ decisions to subcontract with IAMERS members to perform repair work. He further emphasized that the language of the bill would classify any type of repair work performed as a complaint, increasing the burden on third-party services, and hurt rural and regional hospitals that rely heavily on third-party servicers.

Fostering Innovation in Medical Imaging Act 

H.R. 2009 would “provide clarity with respect to the regulation of diagnostic imaging devices intended for use with contrast agents.” Currently, the FDA will not approve imaging devices or enhancements for use with approved contrast agents, if the agents are not specifically labeled for that use. MITA approved of the bill’s provision of “a clear regulatory pathway” to promote innovation.

H.R. 1736

Patricia Shrader, Vice President for Global Regulatory Affairs at Medtronic, Inc., spoke on behalf of AdvaMed, the Advanced Medical Technology Association, to offer its support of the bill, which would establish a risk-based inspection schedule based on a medical device facility’s risk profile. Shrader opined that this would allow the FDA to consider a facility’s compliance history and other related factors in scheduling inspections, as opposed to aligning the frequency of inspections with the classification of the devices a facility manufactures. She applauded the bill’s plan to improve FDA and facility communications prior to, during, and after inspections, noting that facilities would no longer need to wonder from day-to-day, during the course of an ongoing inspection, whether an inspector would be able to travel to the facility. The bill would also require the FDA to provide non-binding feedback on proposed remediation plans, so that facilities are not left wondering what changes they should make. Finally, the bill would require the FDA to implement a process to address refused international device certifications—certificates to foreign governments (CFGs)—when those refusals result from lack of FDA confirmation, rather than issues with the devices.

PDUFA VI reauthorization would aid 21st Century Cures Act implementation

Since 1992, the Prescription Drug User Fee Act (PDUFA) has authorized the FDA to collect user fees from biopharmaceutical manufacturers to supplement Congressional appropriations. Revenues from these fees are used on activities related to the review and regulation of new drug products. In exchange for these fees, the FDA commits to meeting certain performance goals, such as reviewing applications within specified timeframes. The FDA’s ability to collect these fees must be reauthorized every five years. Each five-year reauthorization sets a total amount of fee revenue for the first year and provides a formula for annual adjustments to that total based on inflation and workload changes.

On March 22, 2017, the House Energy and Commerce Committee’s Subcommittee on Health held a hearing to examining the PDUFA program. PDUFA, as reauthorized by the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) (P.L. 112-144), expires in September 2017, and must be reauthorized for the fiscal years 2018 to 2022.

This will be the sixth reauthorization of PDUFA. The proposed agreement (PDUFA VI), builds upon process improvements enacted pursuant to FDASIA, including enhanced support for the Breakthrough Therapy Program. Further, it would aid in the implementation of several key provisions in the 21st Century Cures Act and further streamline the development and review of innovative new drugs for patients. The FDA estimates that the fees negotiated in PDUFA VI will average approximately $1 billion per year.

At the hearing, the following individuals testified on how the program has been implemented to date and presented recommendations pertaining to its reauthorization:

Allen

In his testimony, Allen pointed out that: “Prior to the initial user-fee authorizations, patients in other parts of the world were gaining access to new medicines faster than Americans, with only about 10 percent of new treatments reaching U.S. patients first.” That paradigm has largely been reversed, according to Allen. “Between 2003 and 2016, 73 new cancer drugs were approved by both the FDA and EMA [European Medicines Agency]. Of those drugs, 97 percent (71 of 73) were available in the U.S. before Europe. Furthermore, the FDA approved new cancer drugs on average nearly 6 months faster than the EMA.”

Allen also stated that PDUFA VI:

  • Advances the role of patients and their experiences;
  • supports the continued success of the Breakthrough Therapy Designation, a designation that may be given to a drug intended to treat a serious illness for which preliminary clinical evidence indicates a substantial improvement over any existing interventions. To date, 170 Breakthrough Therapy Designations have been granted, leading to 79 indications approved by the FDA using this process;
  • promotes qualifications and the use of drug development tools;
  • enhances the use of real-world evidence in regulatory decision-making; and
  • effectively communicated scientific advances.

Allen cautioned, however, that “proposed cuts to biomedical research will put the brakes on the engine of discovery, abandon progress on new tools to enhance product evaluation, impede opportunities for new businesses in the biotech sector, and most perilously, jeopardize the development of new medicines for patients desperate for progress.”

Pritchett

In supporting PDUFA VI reauthorization, Pritchett stated: “For nearly twenty-five years, PDUFA has provided much needed resources to the FDA’s human drug review program that has resulted in greater certainty and predictability for patients who depend on safe and effective innovative medicines.” Pritchett also noted the following benefits under PDUFA:

  • The FDA has approved over 1,500 new drugs and biologics since 1992, including treatments for cancer, cardiovascular, neurological, infectious and rare diseases.
  • The number of new medicines being approved on their first review cycle is at a historic high, including approvals for new medicines to treat rare diseases.
  • Review times for drug applications have dropped by nearly 55 percent.
  • The median approval time for standard applications has decreased from 22.1 months in 1993 to an estimated 10 months in 2015.
  • The median approval time for priority applications has similarly decreased from 13.2 months in 1993 to an estimated 7.9 months in 2014.

Pritchett concluded: “At a time when the U.S medical innovation ecosystem is facing severe strains and increased global competition, it is imperative that the FDA is equipped to help us deliver the next generation of new treatments and cures to meet patients’ unmet medical needs. PDUFA VI will help the FDA ensure that patients receive effective and lifesaving drugs, while maintaining the United States’ global leadership in biomedical innovation.”

Holcombe

Holcombe’s testimony cautioned Congress on the cost of the program: “Since 2002, the PDUFA program has grown at an average of 11 percent per year; this is unsustainable moving into the future. Changes are needed that address the fee collection structure to increase efficiency and reduce administrative burdens for both FDA and companies.”

Holcombe believes that the proposed PDUFA VI agreement would address these concerns by:

  • limiting the carryover balance levels, thus reducing possible over-collection of fees and the need for complicated administrative mechanisms to deal with such over-collections;
  • eliminating supplement fees, which will further simplify fee collections;
  • replacing the current product and manufacturing fees with a new program fee that will constitute 80 percent of the annual fee collections; and
  • reducing the percentage that application fees contribute to the total from the current 33 percent to 20 percent, thus mitigating the overall impact of this difficult-to-predict revenue source.

Holcombe also pointed out the benefits of important overlaps between provisions in the 21st Century Cures Act and the proposed PDUFA VI agreement. She offered the following examples of overlap:

  • The 21st Century Cures Act and PDUFA VI are complementary, in terms of ensuring that FDA (1) has and uses effectively an efficient process for qualifying biomarkers; (2) publishes guidance to help applicants for biomarker qualification understand the taxonomy and data standards; (3) makes public a list of qualified biomarkers and pending applications; and (4) engages external experts in biomarker qualification.
  • Patient-focused drug development. Guidance development, public meetings, development of methods and standards for collecting information and data, and use of patient perception and experience information in the FDA regulatory decision about the benefits and risks of a drug are all elements of both 21st Century Cures and the PDUFA VI agreement.
  • Real-world evidence. The 21st Century Cures Act provides helpful context for the work under PDUFA VI, and provisions of the two that differ are easily harmonized.
  • Innovative trial design. While the 21st Century Cures Act focuses on adaptive trials and Bayesian approaches, PDUFA VI takes a broader approach, opening its pilot program to other trial designs while also highlighting adaptive trials and Bayesian approaches.

Holcombe concluded by indicating the Biotechnology Innovation Organization strongly supports and applauds the enactment of 21st Century Cures, and it strongly supports the PDUFA VI proposed agreement.

Woodcock

At the hearing, Subcommittee Vice Chairman Brett Guthrie (R-Ky) asked Woodcock for an update on the FDA’s Oncology Center of Excellence, a key component of 21st Century Cures and a committee-supported initiative. Woodcock elaborated on the center’s structure and the important work it will be doing.

With regards to PDUFA VI, Woodcock noted: “The PDUFA VI reauthorization proposal . . . was submitted to Congress in December under the previous Administration, and reflects a different approach to the federal budget.” She also stated: “Center to PDUFA VI, and its largest single investment component, are plans to elevate patient voices in developing new drugs to treat their diseases. The agreement shares the committee’s goals reflected in the 21st Century Cures Act – and the highest priority of our stakeholders – to leverage essential patient input and insights to fight disease.”